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Spinraza – the first treatment for the rare condition spinal muscular atrophy (SMA) – is set to be available for NHS use for children and adults with SMA Types 1, 2 and 3.

We are delighted to share this news with the SMA community. Without the support and hard work of clinicians, SMA charities, MPs and, above all, families and people with the condition, this would not have been possible. Thanks to all of us working together and our tireless campaigning, patients are facing a brighter future.

What do we know?

NICE, Biogen and NHS England say they have come to an agreement to make the treatment available for children, young people and adults with SMA Types 1, 2 and 3 through a scheme known as a Managed Access Agreement (MAA). This means that patients will be able to get Spinraza while more long-term data on its effectiveness is gathered.

NHS England says the treatment will be made available to the youngest and most severely-affected (SMA Type 1) patients immediately by Biogen, with NHS England offering funding on NICE’s publication of final guidance.

It says that for older babies, children and young adults with SMA Types 2 and 3, the NHS will begin to provide Spinraza shortly after NICE’s guidance is published, once the services to deliver them are established. This is not expected to take more than a few weeks.

You find out more by reading our FAQs on Spinraza.

What happens now?

Muscular Dystrophy UK and Spinal Muscular Atrophy UK are now calling on NICE to formally recommend the treatment and for Biogen and NHS England to urgently implement the MAA so patients can access the drug quickly, and for Wales and Northern Ireland, which generally follows NICE’s guidance, to follow suit. The charities will also continue to push for changes to the appraisal process for rare disease drugs.

In Scotland, Spinraza has been available to SMA Type 1 patients since May 2018, and this is set to be expanded to Types 2 and 3 soon under the Scottish Medicines Consortium’s new ultra-orphan pathway. Meanwhile Spinraza is available for patients with Types 2 and 3 through the Peer Approved Clinical System (PACS) Tier One system for individual requests submitted by clinicians.

Muscular Dystrophy UK will be publishing more information and reaction online and via social media as and when we have it.

Read more at MDUK.